AU2015311681B2 - Globin gene therapy for treating hemoglobinopathies - Google Patents

Globin gene therapy for treating hemoglobinopathies Download PDF

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Publication number
AU2015311681B2
AU2015311681B2 AU2015311681A AU2015311681A AU2015311681B2 AU 2015311681 B2 AU2015311681 B2 AU 2015311681B2 AU 2015311681 A AU2015311681 A AU 2015311681A AU 2015311681 A AU2015311681 A AU 2015311681A AU 2015311681 B2 AU2015311681 B2 AU 2015311681B2
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region
expression cassette
globin
seq
nucleotide sequence
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AU2015311681A1 (en
AU2015311681A8 (en
Inventor
Mingdong LIU
Jorge MANSILLA-SOTO
Isabelle Riviere
Michel Sadelain
George Stamatoyannopoulos
John STAMATOYANNOPOULOS
Xiuyan WANG
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University of Washington
Memorial Sloan Kettering Cancer Center
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University of Washington
Memorial Sloan Kettering Cancer Center
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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AU2015311681A 2014-09-04 2015-09-04 Globin gene therapy for treating hemoglobinopathies Active AU2015311681B2 (en)

Priority Applications (1)

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AU2021290257A AU2021290257A1 (en) 2014-09-04 2021-12-21 Globin gene therapy for treating hemoglobinopathies

Applications Claiming Priority (3)

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US201462045997P 2014-09-04 2014-09-04
US62/045,997 2014-09-04
PCT/US2015/048698 WO2016037138A1 (en) 2014-09-04 2015-09-04 Global gene therapy for treating hemoglobinopathies

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AU2015311681A1 AU2015311681A1 (en) 2017-04-13
AU2015311681A8 AU2015311681A8 (en) 2019-08-08
AU2015311681B2 true AU2015311681B2 (en) 2021-09-23

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US (2) US11717579B2 (zh)
EP (2) EP3189143B1 (zh)
JP (3) JP6710680B2 (zh)
KR (2) KR102500531B1 (zh)
CN (3) CN114457076A (zh)
AU (2) AU2015311681B2 (zh)
BR (1) BR112017004349A2 (zh)
CA (1) CA2960209C (zh)
ES (1) ES2746529T3 (zh)
MX (1) MX2017002900A (zh)
WO (1) WO2016037138A1 (zh)

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AU2015311681A1 (en) 2017-04-13
CN114457076A (zh) 2022-05-10
US20170173185A1 (en) 2017-06-22
EP3189143B1 (en) 2019-08-07
WO2016037138A1 (en) 2016-03-10
EP3620521B1 (en) 2024-05-29
ES2746529T3 (es) 2020-03-06
JP2022107736A (ja) 2022-07-22
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